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Optimized AAV capsids for basal ganglia diseases show robust potency and distribution

19 May 2025

Abstract excerpt

Huntington’s disease and other disorders of the basal ganglia create challenges for biomolecule-based medicines given the poor accessibility of these deep brain structures following intracerebral or intravascular delivery. Here, we found that low dose, low volume delivery of unbiased AAV libraries into the globus pallidus allowed recovery of novel capsids capable of broad access to key deep brain and cortical...

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PMID: 40389462
DOI: 10.1038/s41467-025-60000-3

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